Study of ANAVEX2-73 in Patients With Rett Syndrome

NCT03758924 | Completed Phase 2 FDA Drug

• 1 other identifier: ANAVEX2-73-RS-001
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 8

Brief Summary

Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study. 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.

Conditions

Rett Syndrome

Outcome Measures

Primary Outcomes (4)

• Incidence of Adverse Events

  Incidence of Adverse Events

  7 weeks

• Maximum Plasma Concentration [Cmax] of ANAVEX2-73

  PK of ANAVEX2-73 and metabolite

  7 weeks

• Area Under the Curve [AUC] of ANAVEX2-73

  PK of ANAVEX2-73 and metabolite

  7 weeks

• Lipid panel

  Significant laboratory findings

  7 weeks

Secondary Outcomes (2)

• RSBQ

  7 weeks

• CGI-I

  7 weeks

Other Outcomes (7)

• Anxiety, Depression, and Mood Scale (ADAMS)

  7 weeks

• Children's Sleep Habits Questionnaire (CSHQ)

  7 weeks

• Visual Analog Scale (VAS)

  7 weeks

Study Arms (2)

Active arm

EXPERIMENTAL

Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73)

Drug: ANAVEX2-73

Placebo arm

PLACEBO COMPARATOR

Week 0-7: Take 1 ml orally of the product daily (placebo)

Drug: Placebo

Interventions

ANAVEX2-73 DRUG

Liquid oral solution

Active arm

Placebo DRUG

Liquid oral solution

Placebo arm

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Aged ≥ 18 years, inclusive.
• Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation.
• Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks.
• If on antiepileptic drugs (AEDs), 1-4 AEDs allowed. Treatment must be stable (drug, dose, interval of administration) for 30 days prior to enrollment.
• Ability to keep accurate seizure diaries or have caregiver who can keep accurate seizure diaries.
• Confirmation from the participant that, if of childbearing potential is not pregnant through urine pregnancy testing. Female patients of childbearing potential and at risk for pregnancy must agree to abstinence.
• Prior to the conduct of study-specific procedures, the subject's parent/caregiver/LAR must provide written informed consent. If applicable, the research team must attempt to obtain consent from both parents.

You may not qualify if:

• Patients who have a progressive medical or neurological condition that in the opinion of the Investigator would interfere with the conduct of the study.
• Current clinically significant systemic illness that is likely to result in deterioration of the patient's condition or affect the patient's safety during the study.
• History of clinically evident stroke or clinically significant carotid or vertebrobasilar stenosis or plaque or other history of neurologic (e.g., head trauma with loss of consciousness) or psychiatric condition that the Investigator deems may interfere with interpretability of data.
• Indication of liver disease, defined by serum levels of ALT (SGPT), AST (SGOT), or alkaline phosphatase above 3x upper limit of normal (ULN) as determined during screening.
• Treatment with immunosuppressive medications (e.g., systemic corticosteroids) within the last 90 days (topical and nasal corticosteroids and inhaled corticosteroids for asthma are permitted) or chemotherapeutic agents for malignancy within the last 3 years.
• Other clinically significant abnormality on physical, neurological, laboratory, or electrocardiogram (ECG) examination (e.g., atrial fibrillation) that could compromise the study or be detrimental to the participant.
• Any known hypersensitivity to any of the excipients contained in the study drug or placebo formulation.
• Other co-morbid or chronic illness beyond that known to be associated with RTT.
• Subjects who plan to initiate or change pharmacologic or nonpharmacologic intervention during the course of the study.
• Subjects on potent CYP 3A4 and CYP2C19 inhibitors and inducers.
• Subjects taking another investigational drug currently or within the last 30 days.
• Any other criteria (such as a clinically significant screening blood test result), which in the opinion of the Investigator could interfere with the study conduct or outcome.
• Patients with hepatic and renal impairment.

Sponsors & Collaborators

• Anavex Life Sciences Corp.: lead
• International Rett Syndrome Foundation Rettsyndrome.org: collaborator

Study Sites (8)

UAB | The University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

UC Davis University of California - Davis MIND Institute

Sacramento, California, 95817, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University in St. Louis | Saint Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Greenwood Genetic Center

Greenwood, South Carolina, 29646, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Rett Syndrome

Interventions

tetrahydro-N, N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Study Officials

• Walter Kaufmann, MD

  Emory University SOM

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-Blind, Randomized, Placebo-Controlled
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 31 participants: 6 PK open-label followed by 25 double-blind, randomized, placebo-controlled

Study Record Dates

• First Submitted: November 25, 2018
• First Posted: November 29, 2018
• Study Start: February 28, 2019
• Primary Completion: October 30, 2020
• Study Completion: October 30, 2020
• Last Updated: January 12, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)